The Emerging Era of N-of-1 Drugs for Ultra-Rare Genetic Diseases
Imagine if a medication or therapy could be created to treat just one individual with an ultra-rare disease. There are nearly 7,000 known rare diseases (1) and many are debilitating and ultimately fatal. In the U.S., a disease is considered rare when it affects ...
A Hitchhiker’s Guide to Click-Chemistry with Nucleic Acids
Fantoni NZ, El-Sagheer AH, Brown T. Chem Rev. 2021 Jan 14. Pubmed
MiR-CLIP reveals iso-miR selective regulation in the miR-124 targetome
Wang Y, Soneson C, Malinowska AL, Laski A, Ghosh S, Kanitz A, Gebert LFR, Robinson MD, Hall J. Nucleic Acids Res. 2021 Jan 11;49(1):25-37 Pubmed
A ubiquitin ligase mediates target-directed microRNA decay independently of tailing and trimming
Han J, LaVigne CA, Jones BT, Zhang H, Gillett F, Mendell JT. Science. 2020 Dec 18;370(6523):eabc9546. Pubmed
The ZSWIM8 ubiquitin ligase mediates target-directed microRNA degradation
Shi CY, Kingston ER, Kleaveland B, Lin DH, Stubna MW, Bartel DP. Science. 2020 Dec 18;370(6523):eabc9359. Pubmed
Splicing-associated chromatin signatures: a combinatorial and position-dependent role for histone marks in splicing definition
Agirre E, Oldfield AJ, Bellora N, Segelle A, Luco RF. Nat Commun. 2021 Jan 29;12(1):682. Pubmed
A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis
Krienke C, Kolb L, Diken E, Streuber M, Kirchhoff S, Bukur T, Akilli-Ă–ztĂ¼rk Ă–, Kranz LM, Berger H, Petschenka J, Diken M, Kreiter S, Yogev N, Waisman A, KarikĂ³ K, TĂ¼reci Ă–, Sahin U. Science. 2021 Jan 8;371(6525):145-153. Pubmed
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
Scaglioni D, Catapano F, Ellis M, Torelli S, Chambers D, Feng L, Beck M, Sewry C, Monforte M, Harriman S, Koenig E, Malhotra J, Popplewell L, Guglieri M, Straub V, Mercuri E, Servais L, Phadke R, Morgan J, Muntoni Acta Neuropathol Commun. 2021 Jan 6;9(1):7. ...
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Brain Sci. 2021 Jan 20;11(2):E129. Pubmed .
Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2′-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)
Zanardi TA, Korbmacher B, Boone L, Engelhardt JA, Wang Y, Burel S, Prill B, Aghajan M, Guo S, Henry SP. J Pharmacol Exp Ther. 2021 Jan 11:JPET-AR-2020-000222 Pubmed